EP4356127A4 - Méthodes oligonucléotidiques thérapeutiques - Google Patents

Méthodes oligonucléotidiques thérapeutiques

Info

Publication number
EP4356127A4
EP4356127A4 EP22825838.0A EP22825838A EP4356127A4 EP 4356127 A4 EP4356127 A4 EP 4356127A4 EP 22825838 A EP22825838 A EP 22825838A EP 4356127 A4 EP4356127 A4 EP 4356127A4
Authority
EP
European Patent Office
Prior art keywords
therapeutic oligonucleotide
oligonucleotide methods
methods
therapeutic
oligonucleotide
Prior art date
Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
Pending
Application number
EP22825838.0A
Other languages
German (de)
English (en)
Other versions
EP4356127A2 (fr
Inventor
Caitlin Lewarch
David Gerber
Luis Williams
Duncan Brown
Sudhir Agrawal
Graham T Dempsey
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Quiver Holdings Inc
Original Assignee
Q State Biosciences Inc
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by Q State Biosciences Inc filed Critical Q State Biosciences Inc
Publication of EP4356127A2 publication Critical patent/EP4356127A2/fr
Publication of EP4356127A4 publication Critical patent/EP4356127A4/fr
Pending legal-status Critical Current

Links

Classifications

    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/111—General methods applicable to biologically active non-coding nucleic acids
    • G—PHYSICS
    • G16—INFORMATION AND COMMUNICATION TECHNOLOGY [ICT] SPECIALLY ADAPTED FOR SPECIFIC APPLICATION FIELDS
    • G16B—BIOINFORMATICS, i.e. INFORMATION AND COMMUNICATION TECHNOLOGY [ICT] SPECIALLY ADAPTED FOR GENETIC OR PROTEIN-RELATED DATA PROCESSING IN COMPUTATIONAL MOLECULAR BIOLOGY
    • G16B15/00—ICT specially adapted for analysing two-dimensional [2D] or three-dimensional [3D] molecular structures, e.g. structural or functional relations or structure alignment
    • G16B15/10—Nucleic acid folding
    • G—PHYSICS
    • G16—INFORMATION AND COMMUNICATION TECHNOLOGY [ICT] SPECIALLY ADAPTED FOR SPECIFIC APPLICATION FIELDS
    • G16B—BIOINFORMATICS, i.e. INFORMATION AND COMMUNICATION TECHNOLOGY [ICT] SPECIALLY ADAPTED FOR GENETIC OR PROTEIN-RELATED DATA PROCESSING IN COMPUTATIONAL MOLECULAR BIOLOGY
    • G16B35/00—ICT specially adapted for in silico combinatorial libraries of nucleic acids, proteins or peptides
    • G—PHYSICS
    • G16—INFORMATION AND COMMUNICATION TECHNOLOGY [ICT] SPECIALLY ADAPTED FOR SPECIFIC APPLICATION FIELDS
    • G16B—BIOINFORMATICS, i.e. INFORMATION AND COMMUNICATION TECHNOLOGY [ICT] SPECIALLY ADAPTED FOR GENETIC OR PROTEIN-RELATED DATA PROCESSING IN COMPUTATIONAL MOLECULAR BIOLOGY
    • G16B35/00—ICT specially adapted for in silico combinatorial libraries of nucleic acids, proteins or peptides
    • G16B35/20—Screening of libraries
    • G—PHYSICS
    • G16—INFORMATION AND COMMUNICATION TECHNOLOGY [ICT] SPECIALLY ADAPTED FOR SPECIFIC APPLICATION FIELDS
    • G16H—HEALTHCARE INFORMATICS, i.e. INFORMATION AND COMMUNICATION TECHNOLOGY [ICT] SPECIALLY ADAPTED FOR THE HANDLING OR PROCESSING OF MEDICAL OR HEALTHCARE DATA
    • G16H50/00—ICT specially adapted for medical diagnosis, medical simulation or medical data mining; ICT specially adapted for detecting, monitoring or modelling epidemics or pandemics
    • G16H50/20—ICT specially adapted for medical diagnosis, medical simulation or medical data mining; ICT specially adapted for detecting, monitoring or modelling epidemics or pandemics for computer-aided diagnosis, e.g. based on medical expert systems
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/11—Antisense
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/341—Gapmers, i.e. of the type ===---===
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2320/00—Applications; Uses
    • C12N2320/10—Applications; Uses in screening processes
    • C12N2320/11—Applications; Uses in screening processes for the determination of target sites, i.e. of active nucleic acids
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2330/00—Production
    • C12N2330/30—Production chemically synthesised
    • C12N2330/31—Libraries, arrays

Landscapes

  • Health & Medical Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Biomedical Technology (AREA)
  • Chemical & Material Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Genetics & Genomics (AREA)
  • Physics & Mathematics (AREA)
  • General Health & Medical Sciences (AREA)
  • Biotechnology (AREA)
  • Molecular Biology (AREA)
  • Organic Chemistry (AREA)
  • Biophysics (AREA)
  • Biochemistry (AREA)
  • Medical Informatics (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • Spectroscopy & Molecular Physics (AREA)
  • Public Health (AREA)
  • Evolutionary Biology (AREA)
  • Library & Information Science (AREA)
  • Theoretical Computer Science (AREA)
  • Bioinformatics & Computational Biology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Medicinal Chemistry (AREA)
  • Veterinary Medicine (AREA)
  • Neurology (AREA)
  • Neurosurgery (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Data Mining & Analysis (AREA)
  • Crystallography & Structural Chemistry (AREA)
  • Primary Health Care (AREA)
  • Epidemiology (AREA)
  • Pathology (AREA)
EP22825838.0A 2021-06-17 2022-06-16 Méthodes oligonucléotidiques thérapeutiques Pending EP4356127A4 (fr)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US202163211814P 2021-06-17 2021-06-17
PCT/US2022/033836 WO2022266351A2 (fr) 2021-06-17 2022-06-16 Méthodes oligonucléotidiques thérapeutiques

Publications (2)

Publication Number Publication Date
EP4356127A2 EP4356127A2 (fr) 2024-04-24
EP4356127A4 true EP4356127A4 (fr) 2025-07-09

Family

ID=84490153

Family Applications (1)

Application Number Title Priority Date Filing Date
EP22825838.0A Pending EP4356127A4 (fr) 2021-06-17 2022-06-16 Méthodes oligonucléotidiques thérapeutiques

Country Status (5)

Country Link
US (1) US20220403389A1 (fr)
EP (1) EP4356127A4 (fr)
JP (1) JP2024522763A (fr)
CA (1) CA3224332A1 (fr)
WO (1) WO2022266351A2 (fr)

Families Citing this family (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2025507683A (ja) * 2022-02-24 2025-03-21 キュー－ステート バイオサイエンシーズ， インコーポレイテッド Ｓｙｎｇａｐハプロ不全のための治療剤
TW202535425A (zh) * 2023-12-21 2025-09-16 瑞士商赫孚孟拉羅股份公司 反義寡核苷酸

Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US7517644B1 (en) * 1991-08-23 2009-04-14 Larry J. Smith Method and compositions for cellular reprogramming
WO2015164363A1 (fr) * 2014-04-22 2015-10-29 Q-State Biosciences, Inc. Analyse optogénétique de composés
WO2018165577A1 (fr) * 2017-03-09 2018-09-13 Q-State Biosciences, Inc. Compositions de médiateur de réponse à la douleur et leurs procédés de fabrication et d'utilisation

Patent Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US7517644B1 (en) * 1991-08-23 2009-04-14 Larry J. Smith Method and compositions for cellular reprogramming
WO2015164363A1 (fr) * 2014-04-22 2015-10-29 Q-State Biosciences, Inc. Analyse optogénétique de composés
WO2018165577A1 (fr) * 2017-03-09 2018-09-13 Q-State Biosciences, Inc. Compositions de médiateur de réponse à la douleur et leurs procédés de fabrication et d'utilisation

Non-Patent Citations (1)

* Cited by examiner, † Cited by third party
Title
NAITO YUKI ET AL: "siRNA Design Software for a Target Gene-Specific RNA Interference", FRONTIERS IN GENETICS, vol. 3, 1 January 2012 (2012-01-01), pages 1 - 7, XP055817712, DOI: 10.3389/fgene.2012.00102 *

Also Published As

Publication number Publication date
JP2024522763A (ja) 2024-06-21
CA3224332A1 (fr) 2022-12-22
EP4356127A2 (fr) 2024-04-24
US20220403389A1 (en) 2022-12-22
WO2022266351A3 (fr) 2023-02-16
WO2022266351A2 (fr) 2022-12-22

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Ipc: G16H 50/20 20180101ALI20250530BHEP

Ipc: G16B 35/20 20190101ALI20250530BHEP

Ipc: G16B 15/10 20190101ALI20250530BHEP

Ipc: C12N 15/113 20100101ALI20250530BHEP

Ipc: C12N 15/11 20060101ALI20250530BHEP

Ipc: A61P 25/00 20060101ALI20250530BHEP

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Owner name: QUIVER HOLDINGS INC.